This application relates to a vector comprising a mammalian, and particularly a human globin gene and to the use thereof in treatment of hemoglobinopathies, including α- and β-thalessemia and sickle-cell disease.
Current treatment modalities for β-thalassemias consist of either red blood cell transfusion plus iron chelation (which extends survival but is cumbersome, expensive and an imperfect therapy), or allogeneic bone marrow transplant (which carries a lethal risk and is not available to the majority of patients). Thus, there is a substantial need for improved therapeutic approaches. The present invention provides a genetic correction in autologous hematopoietic stem cells, thus using gene therapy to provide a less-risky and more effective long-term treatment.
While gene therapy has been proposed for many years, a significant challenge facing efforts to develop gene therapy vectors is the ability to produce therapeutically useful levels of a desired protein or peptide. The present invention provides a vector which is capable of providing therapeutically meaningful levels of human globin for sustained periods of time. This ability arises from the ability to transmit large genomic regulatory sequences that control expression of the therapeutic gene.